Becomes first investigational gene therapy to receive ODD for the disease

Dutch biotech UniQure has won orphan drug designation (ODD) for its investigational treatment for Huntington’s Disease, a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioural abnormalities and cognitive decline.

The drug - currently known as AMT-130 - could potentially be the first treatment approved for the disease in the US, with UniQure looking to bring the therapy to clinical trials next year.

This would be a big win for the biotech, as the US Food and Drug Administration (FDA) could offer US market exclusivity for up to seven years thanks to the ODD programme - which provides a ‘special’ status for investigational drugs that are developed for rare disease.

The group published results from preclinical studies last year suggesting that a one-time administration of AAV5-delivered therapy into the central nervous system could block the mutant HTT gene that causes the disease.

According to UniQure, AMT-130 does exactly that as the therapy consists of an AAV5 vector that carries an artificial micro-RNA, which ‘silences’ the Huntington gene when it reaches the brain.

The designation supports UniQure’s “ongoing development in Huntington’s”, which is a therapy area the group has long been interested in, alongside haemophilia and cardiovascular diseases.

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